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Related Concept Videos

CRISPR01:59

CRISPR

58.0K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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CRISPR and crRNAs02:53

CRISPR and crRNAs

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Kidney Structure01:45

Kidney Structure

75.5K
The kidneys are two large bean-shaped organs located in the upper abdomen. They filter the blood several times a day to remove toxins and rebalance water and electrolytes of the circulatory system via the renal veins. The kidneys receive blood directly from the heart via the renal arteries. These arteries enter the kidney at the hilum, the concave surface of the bean, where they branch and divide into smaller vessels and capillaries.
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Gene Flow02:39

Gene Flow

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Gene flow is the transfer of genes among populations, resulting from either the dispersal of gametes or from the migration of individuals.
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CRISPR Gene Editing Tool for MicroRNA Cluster Network Analysis
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CRISPR Gene Editing in the Kidney.

Nelly M Cruz1, Benjamin S Freedman1

  • 1Division of Nephrology, University of Washington School of Medicine, Seattle WA; Kidney Research Institute, University of Washington School of Medicine, Seattle WA; Institute for Stem Cell and Regenerative Medicine, University of Washington School of Medicine, Seattle WA; Department of Medicine, University of Washington School of Medicine, Seattle WA.

American Journal of Kidney Diseases : the Official Journal of the National Kidney Foundation
|April 3, 2018
PubMed
Summary

CRISPR gene editing offers new ways to study and treat kidney diseases by creating disease models and potential therapies. Challenges in delivery and control must be addressed for safe clinical use.

Keywords:
CRISPRanimal modelchimeric antigen receptor T-cells (CAR-T)gene editinggene knockoutgene therapygenomehuman pluripotent stem cells (hPSC)in vitro culturenephrotic syndromeorganoidspolycystic kidney disease (PKD)review

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Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.
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CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.
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Area of Science:

  • Biotechnology
  • Genetics
  • Nephrology

Background:

  • CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a powerful gene editing technology.
  • Kidney diseases present significant health challenges with limited treatment options.

Purpose of the Study:

  • To review the applications of CRISPR gene editing in the study and treatment of kidney diseases.
  • To highlight CRISPR's potential in disease modeling, therapeutic development, and organ transplantation.

Main Methods:

  • Functional genomics experiments using CRISPR in cell lines and model organisms.
  • Development of gene-edited human kidney organoids from pluripotent stem cells.
  • Review of CRISPR-based therapeutic strategies and preclinical models.

Main Results:

  • CRISPR facilitates validation of candidate genes for kidney diseases.
  • Gene-edited kidney organoids provide insights into polycystic kidney disease and nephrotic syndrome.
  • CRISPR-engineered cell therapies show promise for inflammatory kidney conditions and xenotransplantation.

Conclusions:

  • CRISPR technology holds significant potential for advancing kidney disease research and developing novel therapies.
  • Effective delivery and precise control of gene editing remain critical challenges for clinical translation.
  • Further preclinical validation is essential for the safe and effective application of CRISPR in nephrology.